NPS Pharmaceuticals, Inc. (NASDAQ:NPSP), a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases, today announced that the Japanese Ministry of Health, Labor and Welfare (MHLW) has granted teduglutide orphan drug designation for the treatment of Short Bowel Syndrome (SBS). Also, due to the strong medical need for SBS treatments in Japan, the MHLW's expert committee on Unapproved New Drugs and New Indications with High Medical Needs requested that NPS Pharma Japan K.K. develop teduglutide for adult SBS earlier this year. To meet local regulatory requirements, the company is also preparing to initiate a small study in Japanese patients with SBS before the end of this year. "The decision to grant teduglutide orphan drug status in Japan underscores the need for new treatment options for people living with Short Bowel Syndrome," said Roger Garceau, MD, executive vice president and chief medical officer of NPS Pharma. "We look forward to initiating our study and advancing the regulatory process for teduglutide in Japan." In Japan, orphan drug status can be bestowed on drugs or medical devices intended for use in fewer than 50,000 patients for which there is a high medical need. Orphan drugs may be eligible for subsidies and tax credits to reduce the burden of product development, priority consultation and review for marketing authorization, and an extended reexamination period of up to 10 years. The MHLW established the committee on Unapproved New Drugs and New Indications with High Medical Needs to enhance the development of drugs and indications that have been approved for use in the United States and Europe, but are not yet approved in Japan. Due to the strong medical need for SBS treatments in Japan, the committee determined that teduglutide met these criteria and requested that the company develop the drug for adult SBS.
Teduglutide received orphan drug designation for the treatment of SBS from the U.S. Food and Drug Administration in 2000 and the European Medicines Agency in 2001. In the U.S., NPS Pharma markets teduglutide under the trade name Gattex ® and in the EU, as Revestive ®.About Short Bowel Syndrome Short Bowel Syndrome, or SBS, is a rare and potentially fatal gastrointestinal disorder in which patients are unable to absorb enough nutrients and fluids through the intestine from the food they eat to sustain life. SBS typically occurs when a large portion of the intestine has been removed by surgery caused by disease or injury. In rare cases, it is congenital. Common symptoms of SBS can include diarrhea, dehydration, malnutrition, and weight loss, which are closely related to the functional issues of the bowel. Patients with SBS may be dependent on parenteral nutrition, a nutritional supplement administered intravenously, for their survival. About NPS Pharma NPS Pharma is a global biopharmaceutical company pioneering and delivering therapies that transform the lives of patients with rare diseases. The company's current therapeutic areas of focus are gastrointestinal disease and endocrine disorders. These include Short Bowel Syndrome, a potentially fatal gastrointestinal disorder in which patients may have to rely on parenteral nutrition for their survival; Hypoparathyroidism, a complex endocrine disorder in which the parathyroid glands are either absent or damaged, and the body produces insufficient or no parathyroid hormone; and Autosomal Dominant Hypocalcemia, an ultra-rare, genetic disorder of calcium homeostasis caused by mutations of the calcium-sensing receptor gene. NPS Pharma continues to seek in-licensing opportunities to develop new therapies for a broad range of rare diseases, and complements its proprietary programs with a royalty-based portfolio of products and product candidates that includes agreements with Amgen, GlaxoSmithKline, Janssen Pharmaceuticals, and Kyowa Hakko Kirin. NPS Pharma has operations in the U.S., Canada, Europe, Latin America and Japan. Learn more at: www.npsp.com. "NPS Pharma" and "NPS Pharmaceuticals" are the company's trademarks.
Disclosure noticeStatements made in this press release, which are not historical in nature, constitute forward-looking statements for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. These statements are based on the company's current expectations and beliefs and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Forward looking statements include, but are not limited to, statements concerning the company's future financial performance and plans for the commercialization of its products, beliefs or expectations regarding our products in development, statements concerning the company's plans for international expansion, beliefs or expectations regarding potential revenue and earnings from product sales, including beliefs regarding our ability to grow sales, expectations regarding the market size for our products, including those in development, and beliefs or expectations regarding our operating expenses. Risks associated to the company's business include, but are not limited to, the risks associated with any failure by the company to successfully commercialize Gattex/Revestive (teduglutide [rDNA origin]) for injection, including the risk that physicians and patients may not see the advantages of Gattex/Revestive and may therefore be reluctant to utilize the product, the risk that private and public payers may be reluctant to cover or provide reimbursement for Gattex, risks related to regulatory approvals for recombinant human parathyroid hormone 1-84 (rhPTH [1-84]), the risks associated with the company's strategy, global macroeconomic conditions, the impact of changes in management or staff levels, the effect of legislation effecting healthcare reform in the United States, as well as other risk factors described in the company's periodic filings with the U.S. Securities and Exchange Commission, including its Annual Report on Form 10-K and Form 10-Qs. All information in this press release is as of the date of this press release and NPS undertakes no duty to update this information, whether as a result of new information, future events or otherwise.